Temple Uses Gene Editing to Eliminate HIV Infection in Mice

A recent article out of Philadelphia reports that researchers at Temple University had found success using a gene-editing technique to snip out HIV DNA from the genetic code of mice. The findings bolster the hope for an eventual cure for HIV.

The findings are the result of several years of Temple University experiments that initially showed the AIDS virus could be cut from cells in the lab.

University of Pittsburgh scientists, co-led by Temple's Center for Neurovirology's director Kamel Khalili, published the work in the journal Molecular Therapy. The new study is the first to show that HIV replication can be shut down and the virus removed from animal cells via gene editing technology called CRISPR/Cas 9. This technology combines a synthetic "guide RNA -- similar to a 'search function' -- with an enzyme that acts like molecular scissors.

Khalili and his team successfully used this strategy in two mouse models, one representing a newly acquired infection with the virus actively replicating, and the other representing a chronic, latent infection.

The findings are significant because of HIV's elusive reservoir of virus cells. Antiviral drugs suppress replication of these cells so that only a tiny minority of immune cells hold the infection. But if these drugs are stopped, the latent virus in these reservoirs can break out and resume its destructive course.

In a news release, Khalili said the next step would be to repeat the study in primates like monkeys, as they closely mimic human HIV infection. This CRISPR technology is only six years old and has never been used in humans. But the ability to precisely edit genes is seen as a potentially revolutionary medical tool.